Iron oxide-coupled CRISPR-nCas9-based genome editing assessment in mucopolysaccharidosis IVA mice
Author:
Publisher
Elsevier BV
Subject
Genetics,Molecular Biology,Molecular Medicine
Reference78 articles.
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3. Impact of long-term elosulfase alfa treatment on clinical and patient-reported outcomes in patients with mucopolysaccharidosis type IVA: results from a Managed Access Agreement in England;Cleary;Orphanet J. Rare Dis.,2021
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1. CRISPR/Cas9 technology in the modeling of and treatment of mucopolysaccharidosis;Biochemistry and Biophysics Reports;2024-09
2. Nonviral delivery of nCas9 for “safe harbor” integration to treat MPS IVA;Molecular Therapy - Methods & Clinical Development;2024-03
3. Current Strategies for Increasing Knock-In Efficiency in CRISPR/Cas9-Based Approaches;International Journal of Molecular Sciences;2024-02-20
4. Evidence of epigenetic landscape shifts in mucopolysaccharidosis IIIB and IVA;Scientific Reports;2024-02-17
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